mRNA therapy restores ureagenesis and corrects glutathione metabolism in argininosuccinic aciduria
Sonam Gurung, Oskar Vilhelmsson Timmermand, Dany P.B. Perocheau, Ana Luisa Gil-Martínez, Magdalena Minnion, Loukia Touramanidou, Sherry Fang, Martina Messina, Youssef Khalil, Abigail R. Barber, Richard S. Edwards, Patrick F. Finn, Alex Cavedon, Summar Siddiqui, Lisa M. Rice, , Julien Baruteau
This study shows that mRNA therapy restores ureagenesis and glutathione metabolism in ASL-deficient mice, correcting liver disease and neurocognitive impairments.
bioRxiv · Oct 19, 2022 · PreprintGenetics